PSP services: pharma's most effective investment in rare disease care

How PSPs move key metrics and deliver return on investment

In 2023, the pharmaceutical M&A market gradually shook off the effects of the pandemic, with both deal value and volume returning to pre-pandemic levels. Notably, while the number of deals in 2023 slipped about 8% year on year, total deal value grew 37% to surpass US$200 billion. The reason traces back to the pandemic, which stalled negotiations on major transactions for an extended period; with everything now fully reopened, the industry has gravitated toward targets with higher potential value and lower risk, driving deal values upward.

The trend of the big getting bigger is now unmistakable in pharma. By size, the largest deals included Pfizer's US$43 billion acquisition of Seagen, Amgen's US$27.8 billion acquisition of Horizon Therapeutics, Bristol Myers Squibb's US$14 billion acquisition of Karuna Therapeutics and AbbVie's US$10.1 billion acquisition of Immunogen. The Pfizer deal alone illustrates several hallmarks of today's M&A wave: a pharmaceutical giant, ample cash and a target with strong growth potential.

Which therapeutic areas are attracting capital?

From a pharmaceutical company's perspective, investing in rare disease PSPs delivers several tangible business benefits:

• Higher patient retention and loyalty: By helping patients stay on therapy and feel supported, PSPs cultivate loyalty to both the product and the company. Patients who receive emotional, financial and medical support are more likely to continue treatment and trust the brand, building long-term loyalty. AbbVie offers an extensive support program ("Humira Complete") for patients on Humira (which, while not a rare disease drug, treats complex immune conditions). Studies of the program show concrete benefits: participating patients improved adherence by roughly 29% at 12 months and maintained better adherence over 2–3 years, with a 30% lower risk of discontinuation than non-participants and significantly fewer hospitalizations. AbbVie uses these results to demonstrate that its patient services improve real-world outcomes and patient retention. The case is frequently cited as evidence that PSPs can drive health and business outcomes at the same time (longer treatment duration per patient).
• Broader market penetration and access: PSPs effectively lift market penetration by removing barriers to treatment initiation. They provide insurance navigation, financial assistance and education, increasing the share of new patients who start therapy. Metrics such as "shorter time to first dose" and more patients staying on treatment demonstrate the PSP's value to prescribers. Physicians are also more inclined to prescribe drugs that come with patient support, knowing patients will receive more complete care beyond the clinic. Sanofi's programs for certain rare diseases include free counselor services for patients and families, which is especially important for caregivers navigating pediatric rare disease care. This psychosocial support raises patient satisfaction and can indirectly improve adherence (families who feel supported are more likely to stick with demanding treatment regimens). It also differentiates Sanofi's products by underscoring the company's commitment to patients' overall well-being — not just drug supply.
• Data generation and real-world evidence (RWE): PSPs generate a wealth of real-world data on how patients use medicines, adherence patterns, outcomes achieved and reasons for discontinuation. This real-world data (RWD) is enormously valuable to pharmaceutical companies. Companies can analyze PSP data to understand patient needs and treatment performance outside clinical trial settings. That, in turn, supports better evidence for regulators and payers: long-term outcome data collected through PSPs can, for example, support regulatory approval of new indications or satisfy post-marketing study requirements. Several companies have already integrated RWE collection into their PSPs — for instance, PSP vendors pair patient support with electronic health record data, continuously collecting outcomes with patient consent. For manufacturers, this rich real-world evidence can inform ongoing development (drug lifecycle management) and strengthen the case for their therapies' effectiveness in practice.
• Higher patient satisfaction and brand reputation: A well-run PSP dramatically improves the patient's treatment experience, which in turn lifts the company's reputation. High patient satisfaction (often measured through surveys or NPS) signals that the pharmaceutical company delivers value beyond the drug itself. Such positive experiences make patients more likely to continue treatment and champion the therapy within patient communities. In rare diseases, word of mouth and community advocacy carry enormous weight; pharmaceutical companies that invest in patient-centered services can position their brands and products as patient-friendly. That goodwill can translate into stronger relationships with patient advocacy groups and even regulators, who increasingly encourage patient-centered drug development.
• Better alignment with value-based models: As healthcare shifts toward value-based care, pharmaceutical companies need to prove their therapies genuinely improve outcomes cost-effectively. PSPs help here by improving drug-related real-world outcomes (adherence, complications avoided). Better adherence means payers see better value from expensive rare disease therapies. In practice, improving treatment adherence through a PSP "strengthens the product's value proposition" and reduces payers' uncertainty about outcomes. Some innovative arrangements tie PSPs directly to value-based contracts. Globally, for example, there is a model in which the manufacturer provides financial relief if patients on an expensive therapy fail to achieve expected outcomes (outcome-based rebates). Such contracts are only feasible with robust patient support and monitoring to track outcomes. By investing in PSPs, pharmaceutical companies signal services aligned with value-based care goals. In one case, Novartis obtained clearance from the US Office of Inspector General (OIG) to provide travel and lodging support for low-income patients receiving its CAR-T gene therapy (Kymriah), improving access while staying compliant. Such support not only helps patients but also demonstrates to policymakers and payers the company's commitment to outcomes (ensuring patients can actually receive treatment safely and be followed long term).

The strategic advantages PSPs deliver

Beyond immediate metrics and ROI, PSPs give pharmaceutical companies broader strategic advantages in rare diseases:

• Product differentiation: In competitive therapeutic areas (including rare diseases with multiple treatment options), a strong PSP can make a product stand out. A well-designed PSP "demonstrates the product's value beyond standard efficacy and safety measures." Indeed, payers treat PSP services as a meaningful differentiator when evaluating similar drugs. By genuinely meeting patient needs (education, financial assistance, care coordination), a PSP turns a therapy into more than a drug — it becomes a comprehensive solution. That can shape physicians' perception of a company's commitment to patient care and tilt them toward its products. Takeda and Sanofi, for example, have distinguished their enzyme replacement therapies in rare genetic diseases by providing extensive patient support (home injections, disease education and more), making their products more attractive to providers and patients. Researchers and industry experts note that patient-centered PSPs are a "win for all parties," improving outcomes while adding value to the pharmaceutical company's product.
• Faster market penetration: The launch phase of a rare disease drug can be improved through hub services that streamline onboarding for new patients. Faster patient enrollment verification, prior authorization support and patient education delivered through PSPs let treatment start sooner after launch. That means a new therapy gains a foothold in the market faster. Moreover, as noted, physicians are more comfortable prescribing new rare disease drugs when the support infrastructure exists (they know patients will get help with complex self-injection, side effect management and more). In Asia, where rare disease awareness may be lower and health systems more fragmented, PSPs can likewise accelerate penetration by bridging gaps. Companies like Takeda have used PSPs in Asia (for example, in Southeast Asia, through partnerships with NGOs, patient groups or specialist advisors) to raise diagnosis rates and get eligible patients onto treatment, expanding the treatable market.
• Stronger stakeholder relationships: Patient support programs can improve pharmaceutical companies' relationships with key stakeholders — regulators, payers and providers. Regulators see value in programs that ensure safe use and monitoring (some programs require risk evaluation and mitigation strategies; REMS, which typically include a patient education component, are effectively a form of PSP). Better adherence data reduces payers' uncertainty and strengthens the drug's value argument. When a manufacturer can show through its PSP data that "patients on our therapy stay on treatment longer and achieve better outcomes," it strengthens its hand in pricing and reimbursement negotiations. Providers (healthcare professionals) benefit too: PSPs handle ancillary matters (insurance paperwork, patient training), easing the burden on medical teams and making healthcare professionals more willing partners.

Regional barriers and efforts in PSP development

United States: In the US, PSPs (often called "hubs" for specialty drugs) are an established component of rare disease product strategy. They are designed to navigate the complex reimbursement environment and ensure patients do not drop off treatment due to financial or logistical barriers. Utilization, however, remains far from maximized — surveys find low patient awareness and participation (only about 3–8% of eligible patients use these programs, mainly for lack of awareness). Pharmaceutical companies are working to close that gap by promoting PSPs more actively through physicians, pharmacists and digital channels. Compliance and regulatory scrutiny in the US are strict: companies must design PSPs carefully to avoid being deemed inducements (certain co-pay assistance, for example, is restricted in federal programs). Despite these challenges, the trend is for patient support to become integral to rare disease therapy launches, with companies like Pfizer creating dedicated rare disease support platforms (such as Pfizer's "Together for Rare" and the VyndaLink program for tafamidis patients) to connect patients with the services they need. A well-run PSP can improve treatment persistence. AbbVie's Humira® case illustrates this: by providing nursing support, education and follow-up care, the company achieved measurable 1–3 year adherence improvements among patients enrolled in its PSP, with significantly fewer hospitalizations than non-participants.

Asia: In Asia, PSP deployment is accelerating, though maturity varies by country. In Japan and South Korea, many global pharmaceutical companies expand their patient support services (education, call centers and so on) at product launch, much as in Western markets. In emerging Asian markets, PSPs are a newer concept but gaining traction, typically focused on access and affordability. India, for example, now offers PSPs in oncology, rare genetic diseases and other specialties. Because 70% of India's market is out-of-pocket, PSPs there mainly provide financial counseling, free drugs or co-pay assistance, and patient education. Gaps remain: many programs are urban-centric and fragmented, with limited rural coverage and language barriers. Awareness is also an issue in Asian markets — unlike in the US, where PSP information is public and officially accessible, PSP information in Asian countries is very hard to find. The most open data currently comes from China, which has begun encouraging patient support programs to improve access to rare disease drugs (for example, through charitable foundations or tiered pricing schemes), while Southeast Asian countries are discussing frameworks for compassionate use and PSP-like models.

In Taiwan, PatientsForce has localized PSP services for major international pharmaceutical companies, covering insurance navigation, financial assistance, patient education, nursing support and medication adherence coaching — even linking into home healthcare and home nursing systems. Measured by return on investment, treatment adherence among the rare disease patients it serves improves significantly, while healthcare institutions are relieved of staffing burdens, raising patients' satisfaction with their care.